NEI’s investment in vision research ensures that new tools and therapies are shared with the broader scientific community so other labs can build on the discoveries, accelerating progress for patients everywhere.
These advances have the potential to extend beyond eye diseases, offering new hope for conditions affecting the brain, heart, and other tissues.
“The tools that we're developing for ocular gene therapy can be applied outside the eye, too, and so you can see a scenario where that's not just useful in the retina, but it could be useful in the brain and the heart and various tissues of the body.”
With NEI support spanning more than a decade, Dr. Shannon Boye, a genetic researcher at the University of Florida, has been pioneering gene therapy approaches that target the root causes of vision loss, many of which can lead to severe childhood blindness.
Dr. Boye’s research focuses on engineering harmless viruses called adeno-associated viruses (AAV) to deliver healthy genes directly to the cells in the eye that need them. These therapeutic “taxi cabs” don’t just treat symptoms; they target the root cause of vision loss. Dr. Boye’s work addresses major barriers to gene therapy, such as immune responses and the challenge of reaching key parts of the eye without damaging delicate retinal tissue.
“There are no secrets in science—when you discover tools with NIH funding, they are shared with the broader scientific community. That results in a rising tide that lifts all ships and leads to more treatments for more patients.”
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A Legacy of Innovation
The first FDA-approved gene therapy, Luxturna was developed based on intramural research led by NEI scientists.